NCT02601937

EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma

A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 109 (actual)
• Sponsor: Epizyme, Inc. · Industry
• Sex: All
• Age: 6 Months – 17 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase I, open-label, dose escalation and dose expansion study with BID (suspension) and TID (tablet) oral dose of the enhancer of zeste homolog-2 (EZH2) inhibitor, tazemetostat. Subjects will be screened for eligibility within 14 days of the planned first dose of tazemetostat. A treatment cycle will be 28 days. Response assessment will be evaluated after 8 weeks of treatment and subsequently every 8 weeks while on study.

Detailed description

The study has two parts: Dose Escalation and Dose Expansion. Dose escalation for subjects with the following relapsed/refractory malignancies: * Rhabdoid tumors: * Atypical teratoid rhabdoid tumor (ATRT) * Malignant rhabdoid tumor (MRT) * Rhabdoid tumor of kidney (RTK) * Selected tumors with rhabdoid features * Integrase interactor 1 (INI1)-negative tumors: * Epithelioid sarcoma * Epithelioid malignant peripheral nerve sheath tumor * Extraskeletal myxoid chondrosarcoma * Myoepithelial carcinoma * Renal medullary carcinoma * Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) * Synovial Sarcoma with a SS18-SSX rearrangement Dose Escalation cohorts are closed to enrollment. Dose Expansion at the maximum tolerated dose (MTD) or the recommended phase 2 dose (RP2D) * Cohort 1 - ATRT * Cohort 2 - MRT/RTK/selected tumors with rhabdoid features * Cohort 3 - INI-negative tumors: * Epithelioid sarcoma * Epithelioid malignant peripheral nerve sheath tumor * Extraskeletal myxoid chondrosarcoma * Myoepithelial carcinoma * Renal medullary carcinoma * Chordoma (poorly differentiated or de-differentiated) * Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) * Cohort 4 - Tumor types eligible for Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement

Conditions

• Rhabdoid Tumors
• INI1-negative Tumors
• Synovial Sarcoma
• Malignant Rhabdoid Tumor of Ovary

Interventions

Type	Name	Description
DRUG	Tazemetostat	Tazemetostat (EPZ-6438) is a selective small molecule inhibitor of the histone-lysine methyltransferase EZH2 gene.

Timeline

• Start date: 2016-01-07
• Primary completion: 2021-06-19
• Completion: 2021-10-22
• First posted: 2015-11-11
• Last updated: 2024-10-03
• Results posted: 2024-10-03

Locations

35 sites across 9 countries: United States, Australia, Canada, Denmark, France, Germany, Italy, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study